Toni Cathomen

CRISCross: Integration of Epigenetic Information for Deep-Learning-Based Prediction of CRISPR-Cas9 Off-Targets Across Cell Types

CRISCross improves CRISPR-Cas9 off-target prediction by integrating epigenetic features with sequence data across cell types, showing better performance than sequence-only models.

CD117 epitope-shielded hematopoietic stem cell transplantation with toxin-free conditioning and in vivo selection ameliorates β-thalassemia model

This paper demonstrates a method using CD117-blocking antibody and epitope-engineered HSPCs to treat β-thalassemia without myeloablative conditioning.

Clinical development of gene edited tacrolimus-resistant Treg (FKBP12KO-Treg) to enable simultaneous immunosuppression and support of immune regulation

This paper develops gene-edited tacrolimus-resistant Treg cells (FKBP12KO-Treg) for clinical use in transplant recipients, showing they maintain immunoregulatory function while resisting tacrolimus.

Next-generation all-in-one CRISPR/Cas9 multiply-edited CD30CAR-T cells:Potency despite risk of translocations

This study develops next-generation CD30CAR-T cells using CRISPR/Cas9 for multiplex editing, showing high potency but risks of translocations with combined knockouts.

Castling, a novel therapeutic concept for rewiring pathological gene-expression networks, enabled by the TRIPLE technology

The paper introduces a therapeutic strategy called castling to correct gene-expression imbalances by rewiring miRNA regulation using the TRIPLE genome-editing technology.

Repurposing base editors for targeted knock-in and simultaneous knockouts to generate multiplex-edited allogeneic CAR T cells with minimal translocations

This paper develops BEKI, a base editor-based method for targeted transgene insertion and multiplex gene disruption in T cells, reducing chromosomal translocations compared to Cas9-based approaches.

Gene editing of CD3 epsilon gene to redirect regulatory T cells for adoptive T cell transfer

This paper develops a non-viral CRISPR-Cas12a method to redirect Tregs with CARs by integrating an antigen-binding domain into the CD3 epsilon gene, showing improved antigen-specific function and graft protection in a mouse model.

Validation and Long-Term Follow Up of CD33 Off-Targets Predicted In Vitro and In Silico Using Error-Corrected Sequencing in Rhesus Macaques

The paper validates off-target effects of CRISPR/Cas9 in rhesus macaques using error-corrected sequencing, showing that in vitro and in silico methods have limited predictive accuracy.

Therapeutic gene editing in hematopoietic progenitor cells from a mouse model of Fanconi anemia

This paper demonstrates efficient gene editing in FA mouse models using TALENs and a donor template at a safe harbor locus, achieving phenotypic correction of FA cells.